Fujiwara Hideaki, Maeda Yoshinobu, Sando Yasuhisa, Nakamura Makoto, Tani Katsuma, Ishikawa Tatsunori, Nishimori Hisakazu, Matsuoka Ken-Ichi, Fujii Nobuharu, Kondo Eisei, Tanimoto Mitsune
Department of Hematology, Oncology and Respiratory Medicine, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama, Japan.
Transfusion. 2016 Apr;56(4):886-92. doi: 10.1111/trf.13437. Epub 2015 Dec 29.
Transplant-associated thrombotic microangiopathy (TA-TMA) after hematopoietic stem cell transplantation (HSCT) remains a severe complication associated with underlying endothelial damage. TMA has a high mortality rate with no definite treatments and effective treatments are needed.
The study objective was to retrospectively analyze the outcome of patients receiving recombinant human soluble thrombomodulin (rTM), which has cytoprotective effects against calcineurin inhibitor-induced endothelial cell damage, or other therapeutics for TA-TMA from 254 consecutive HSCT recipients between 2009 to 2014 at a single institution. We hypothesized that patients receiving rTM as a first-line treatment would receive a benefit.
Sixteen patients were diagnosed as TA-TMA. Of these 16 patients, nine were treated with rTM (rTM group), and seven received treatment other than rTM (control group) as a first-line therapy. Seven of the nine patients in the rTM group recovered from TA-TMA without complications, but none in the control group recovered. The rTM group showed a significantly better overall survival after TA-TMA onset than did the control group (median, 123.0 days vs. 45.5 days, respectively; p = 0.045). The cumulative incidence of acute graft-versus-host disease was the same in both groups (56% vs. 57%, respectively; p = 0.52) on Day 100 after TA-TMA onset.
This is the first report evaluating rTM administration for TA-TMA compared with previous treatments. Our data suggests that rTM might offer a better clinical outcome in patients with TA-TMA.
造血干细胞移植（HSCT）后移植相关血栓性微血管病（TA-TMA）仍然是一种与潜在内皮损伤相关的严重并发症。TA-TMA死亡率高，尚无明确治疗方法，因此需要有效的治疗手段。
本研究旨在回顾性分析2009年至2014年间在单一机构接受连续254例HSCT患者中，接受具有细胞保护作用、可对抗钙调神经磷酸酶抑制剂诱导的内皮细胞损伤的重组人可溶性血栓调节蛋白（rTM）或其他TA-TMA治疗方法的患者的预后情况。我们假设接受rTM作为一线治疗的患者会从中获益。
16例患者被诊断为TA-TMA。在这16例患者中，9例接受了rTM治疗（rTM组），7例接受了rTM以外的其他治疗（对照组）。rTM组的9例患者中有7例从TA-TMA中康复且无并发症，而对照组无一例康复。TA-TMA发病后，rTM组的总生存率显著高于对照组（中位数分别为123.0天和45.5天；p = 0.045）。TA-TMA发病后第100天，两组急性移植物抗宿主病的累积发生率相同（分别为56%和57%；p = 0.52）。
这是第一篇评估rTM与以往治疗方法相比用于TA-TMA治疗的报告。我们的数据表明，rTM可能为TA-TMA患者带来更好的临床结果。
